Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

• Published in: Journal of comparative effectiveness research Vol. 9; no. 5; pp. 341 - 360
• Main Authors: Mercuri, Eugenio, Muntoni, Francesco, Osorio, Andrés Nascimento, Tulinius, Már, Buccella, Filippo, Morgenroth, Lauren P, Gordish-Dressman, Heather, Jiang, Joel, Trifillis, Panayiota, Zhu, Jin, Kristensen, Allan, Santos, Claudio L, Henricson, Erik K, McDonald, Craig M, Desguerre, Isabelle
• Format: Journal Article
• Language: English
• Published: England Future Medicine Ltd 01.04.2020
• Subjects: Age; ataluren; Blood pressure; Clinical trials; Codon, Nonsense - genetics; Data collection; dystrophin; Dystrophin - genetics; effectiveness; Enrollments; Humans; Hypertension; Medical laboratories; Muscular Dystrophy, Duchenne - drug therapy; Muscular Dystrophy, Duchenne - genetics; Mutation; Neurosciences; Neurovetenskaper; nonsense mutation Duchenne muscular dystrophy; Oxadiazoles - adverse effects; Oxadiazoles - therapeutic use; Patients; Proteins; Registries; safety; STRIDE Registry; Studies; Treatment Outcome; effectiveness; nonsense mutation Duchenne muscular dystrophy; dystrophin; STRIDE Registry; safety; ataluren
• ISSN: 2042-6305; 2042-6313; 2042-6313
• DOI: 10.2217/cer-2019-0171

Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype–phenotype/–ataluren benefit correlations and ataluren safety. Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Kaplan–Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p ≤ 0.05). There were no DMD genotype–phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. NCT02369731. NCT02369731.